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A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

Primary Purpose

Fabry Disease

Status
Completed
Phase
Phase 4
Locations
Japan
Study Type
Interventional
Intervention
Agalsidase beta
Sponsored by
Genzyme, a Sanofi Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fabry Disease focused on measuring cardiac fabry disease

Eligibility Criteria

20 Years - 64 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria) In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants) Left ventricular hypertrophy was noted. Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was related by birth.) Without symptoms or signs of Fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign. Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent. Participants classification: inpatients and outpatients Participants who had given written informed consent before the study-related baseline tests. Exclusion Criteria: Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication) Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent. Participants who had undergone kidney transplantation or were currently on dialysis. Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit). Permanent pacemaker or defibrillator implanted participants Pregnant or lactating women Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent. Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent. Enzyme replacement therapy history, except for agalsidase beta Participants who were unwilling to comply with the requirements of the protocol. Others judged by the investigator or sub-investigator to be ineligible for the study

Sites / Locations

  • Fujita Health University Hospital
  • Sapporo Medical University Hospital
  • Akune Citizen Hospital
  • Tohoku University Hospital
  • Nihon University Itabashi Hospital
  • Nihon University Nerima Hikarigaoka Hospital
  • Yamanashi Prefectural Central Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Agalsidase Beta

Arm Description

Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.

Outcomes

Primary Outcome Measures

Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
Left ventricular mass was assessed by echocardiogram.
Change From Baseline in LVM at Week 156
Left ventricular mass was assessed by echocardiogram.

Secondary Outcome Measures

Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test
Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
Percent Change From Baseline in GL-3 Plasma Levels at Week 156
Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156
The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).

Full Information

First Posted
August 30, 2005
Last Updated
April 16, 2015
Sponsor
Genzyme, a Sanofi Company
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1. Study Identification

Unique Protocol Identification Number
NCT00140621
Brief Title
A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease
Official Title
A Multicenter Open-label Study of the Safety and Efficacy of α-galactosidase A (R-h α-GAL) Replacement Therapy in Patients With Cardiac Fabry Disease
Study Type
Interventional

2. Study Status

Record Verification Date
April 2015
Overall Recruitment Status
Completed
Study Start Date
July 2005 (undefined)
Primary Completion Date
August 2012 (Actual)
Study Completion Date
August 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genzyme, a Sanofi Company

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
This is a multi-center, open label, phase IV study conducted to evaluate the efficacy and safety of agalsidase beta (Fabrazyme [recombinant form]) administered by intravenous drip infusion in participants with cardiac Fabry disease. Participants participated for 4 weeks or less in the baseline period and 156 weeks for the treatment period.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fabry Disease
Keywords
cardiac fabry disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
6 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Agalsidase Beta
Arm Type
Experimental
Arm Description
Agalsidase beta 1 milligram per kilogram (mg/kg) intravenously once every 2 weeks up to 156 weeks.
Intervention Type
Drug
Intervention Name(s)
Agalsidase beta
Other Intervention Name(s)
Fabrazyme®
Primary Outcome Measure Information:
Title
Percent Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Description
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Time Frame
Baseline to Week 156
Title
Change From Baseline in Interventricular Septum and Left Ventricular Posterior Wall Thickness at Week 156
Description
Interventricular septum and left ventricular posterior wall thickness was assessed by echocardiogram.
Time Frame
Baseline to Week 156
Title
Percent Change From Baseline in Left Ventricular Mass (LVM) at Week 156
Description
Left ventricular mass was assessed by echocardiogram.
Time Frame
Baseline to Week 156
Title
Change From Baseline in LVM at Week 156
Description
Left ventricular mass was assessed by echocardiogram.
Time Frame
Baseline to Week 156
Secondary Outcome Measure Information:
Title
Number of Participants in Overall Cardiac Function Assessment and Clinical Symptoms at Week 156: Change From Baseline in Cardiac Function Test
Description
Overall cardiac function assessment was assessed by tests (echocardiogram,cardiac catheterization (optional),electrocardiogram,B-type natriuretic peptide [BNP]), clinical symptoms (subjective symptoms) and the New York Heart Association (NYHA) cardiac functional classification.Overall assessment of cardiac function was assessed based on the evaluation items including interventricular septum thickness, left ventricular posterior wall thickness, left ventricular mass, clinical function tests and clinical symptoms. A subject was considered to be Improved: if Improved in 2 items or more, Unchanged: Improved in one item and unchanged in 2 items or unchanged in all 3 items, Aggravated: Aggravated in one item or more.
Time Frame
Baseline to Week 156
Title
Percent Change From Baseline in GL-3 Plasma Levels at Week 156
Time Frame
Baseline to Week 156
Title
Change From Baseline in Short Form (36) Health Survey (SF-36) Scores at Week 156
Description
The 36-Item Short-Form Health Survey (SF-36) is a standardized survey evaluating 8 aspects of functional health and well-being: physical and social functioning, physical and emotional role limitations, bodily pain, general health, vitality, mental health. These 8 aspects can also be summarized as physical component score (PCS) and mental component score (MCS). The score for a section is an average of the individual question scores, which are scaled 0-100 (100=highest level of functioning).
Time Frame
Baseline to Week 156

10. Eligibility

Sex
All
Minimum Age & Unit of Time
20 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participants definitively diagnosed with cardiac Fabry disease (who fulfill all of the following criteria) In the case of male participants, documented plasma or leukocyte alpha-galactosidase A (α-GAL) activity was no more than 20 percent (%) of normal value (except for heterozygous female participants) Left ventricular hypertrophy was noted. Accumulation of globotriaosylceramide (GL-3) in the myocardium or a genetic deficiency associated with α-GAL was confirmed Or in the case of heterozygous female participants, when the family (father or son) was diagnosed with Fabry disease. (Father or son was related by birth.) Without symptoms or signs of Fabry, such as acroparesthesia, angiokeratomas, abnormal sweating, pain of distal extremities, chronic abdominal pain/diarrhea and corneal opacities were observed, except for proteinuria sign. Participants with interventricular and posterior wall thickness of at least 13 millimeter (mm) on echocardiography within 3 months before signed date to informed consent Participants in whom cardiac function was rated as Class I or II according to the New York Heart Association (NYHA) classification when giving informed consent. Participants classification: inpatients and outpatients Participants who had given written informed consent before the study-related baseline tests. Exclusion Criteria: Participants with severe hypertension (for example, blood pressure more than or equal to 180 millimeter of mercury [mmHg] and/or blood pressure more than or equal to 110 mmHg in spite of adequate medication) Participants whose serum creatinine level was higher than the upper normal limit within 3 months (12 weeks) prior to giving informed consent. Participants who had undergone kidney transplantation or were currently on dialysis. Participants with any serious hepatic disorder. Participants who had abnormal hepatic function test values within 3 months (12 weeks) prior to giving informed consent (when either alanine aminotransferase [ALT] or aspartate aminotransferase [AST] level exceeded the value five times as high as the upper normal limit). Permanent pacemaker or defibrillator implanted participants Pregnant or lactating women Participants who had taken this drug for 6 months (26 weeks) or more before giving informed consent. Participants who had participated in a clinical study employing any other investigational product within 3 months prior to giving informed consent. Enzyme replacement therapy history, except for agalsidase beta Participants who were unwilling to comply with the requirements of the protocol. Others judged by the investigator or sub-investigator to be ineligible for the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Monitor
Organizational Affiliation
Genzyme, a Sanofi Company
Official's Role
Study Director
Facility Information:
Facility Name
Fujita Health University Hospital
City
Aichi
ZIP/Postal Code
470-1192
Country
Japan
Facility Name
Sapporo Medical University Hospital
City
Hokkaido
ZIP/Postal Code
060-8543
Country
Japan
Facility Name
Akune Citizen Hospital
City
Kagoshima
ZIP/Postal Code
899-1611
Country
Japan
Facility Name
Tohoku University Hospital
City
Miyagi
ZIP/Postal Code
980-8574
Country
Japan
Facility Name
Nihon University Itabashi Hospital
City
Tokyo
ZIP/Postal Code
173-8610
Country
Japan
Facility Name
Nihon University Nerima Hikarigaoka Hospital
City
Tokyo
ZIP/Postal Code
179-0072
Country
Japan
Facility Name
Yamanashi Prefectural Central Hospital
City
Yamanashi
ZIP/Postal Code
400-8506
Country
Japan

12. IPD Sharing Statement

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A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

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